Figure 4: siRNA-mediated repression of Htt does not alter its somatic instability or transcriptional rate in HttQ111/+ mice.
A) Schematic of study design. B) Chronic (10 mg/kg/month) siRNA treatment reduces levels of both wildtype (74% reduction; p < 0.0001) and mutant HTT (55% reduction; p < 0.0001) in the liver, as assayed with MSD at 6 months of age following 3.5 months of treatment (N = 10 per arm). C) Total Htt mRNA, as assayed by mouse Htt qRT-PCR, is also reduced by siRNA treatment (64% reduction; N = 10, p < 0.0001). D) Htt pre-mRNA levels are not impacted by siRNA treatment (NS). E) Chronic siRNA treatment does not change SI in HTT’s CAG tract (NS).